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1516 Part IX Cell-Based Therapies
The next frontier of cellular therapies is being driven by the dis- 2. Fischer A, Hacein-Bey Abina S, Touzot F, et al: Gene therapy for primary
covery and ability to culture stem cell populations from various other immunodeficiencies. Clin Genet 88(6):507–515, 2015.
adult tissues (retina, cornea, heart, lung, etc.), embryonic stem cells, 3. Naldini L: Gene therapy returns to centre stage. Nature 526(7573):351–
and inducible pluripotent stem cells. The therapeutic application of 360, 2015.
these cell populations, although intensely investigated worldwide, is 4. Cheever MA, Higano CS: PROVENGE (Sipuleucel-T) in prostate
regarded as preliminary at present and guidelines have been published cancer: the first FDA-approved therapeutic cancer vaccine. Clin Cancer
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for clinical development. Similarly, gene editing is also being used Res 17(11):3520–3526, 2011.
to modify hematopoietic stem cells and immune effector cells in 5. June CH, Riddell SR, Schumacher TN: Adoptive cellular therapy: a race
preclinical studies, but there are ethical considerations with transfer to the finish line. Sci Transl Med 7(280):280ps7, 2015.
to the clinic. 8 6. Horowitz MM, Confer DL: Evaluation of hematopoietic stem cell
donors. Hematology Am Soc Hematol Educ Program 469–475, 2005.
7. Kimmelman J, Hyun I, Benvenisty N, et al: Policy: Global standards for
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8. Kohn DB, Porteus MH, Scharenberg AM: Ethical and regulatory aspects
1. Weiden PL, Flournoy N, Thomas ED, et al: Antileukemic effect of of genome editing. Blood 127(21):2553–2560, 2016.
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